4DMT Presents Positive 60-Week Results from 4D-150 SPECTRA Clinical Trial in DME and Regulatory Update
4D Molecular Therapeutics (NASDAQ:FDMT) announced positive 60-week results from the SPECTRA clinical trial evaluating 4D-150 in diabetic macular edema (DME) patients. The Phase 3 dose (3E10 vg/eye) demonstrated significant efficacy with a 78% reduction in treatment burden compared to projected aflibercept treatment.
Key results include sustained visual acuity gains of +9.7 letters and anatomic improvement with CST reduction of -174 碌m. The treatment was well-tolerated with no intraocular inflammation observed. Notably, patients receiving the Phase 3 dose required only 1.6 mean supplemental injections compared to 3.7 for lower doses and 7.0 projected for aflibercept.
Both FDA and EMA have aligned that a single Phase 3 clinical trial would be acceptable for regulatory submission for 4D-150 in DME, streamlining the path to potential approval.
4D Molecular Therapeutics (NASDAQ:FDMT) ha annunciato risultati positivi a 60 settimane dallo studio clinico SPECTRA che valuta 4D-150 in pazienti con edema maculare diabetico (DME). La dose di Fase 3 (3E10 vg/occhio) ha mostrato un'efficacia significativa con una riduzione del 78% del carico di trattamento rispetto al trattamento previsto con aflibercept.
I risultati chiave includono guadagni sostenuti di acuit脿 visiva di +9,7 lettere e un miglioramento anatomico con una riduzione dello spessore della retina centrale (CST) di -174 碌m. Il trattamento 猫 stato ben tollerato senza infiammazione intraoculare osservata. In particolare, i pazienti trattati con la dose di Fase 3 hanno richiesto in media solo 1,6 iniezioni supplementari rispetto a 3,7 per dosi inferiori e 7,0 previste per aflibercept.
Sia la FDA che l'EMA hanno concordato che un unico studio clinico di Fase 3 sarebbe sufficiente per la presentazione regolatoria di 4D-150 nel DME, semplificando il percorso verso una possibile approvazione.
4D Molecular Therapeutics (NASDAQ:FDMT) anunci贸 resultados positivos a las 60 semanas del ensayo cl铆nico SPECTRA que eval煤a 4D-150 en pacientes con edema macular diab茅tico (DME). La dosis de Fase 3 (3E10 vg/ojos) demostr贸 una eficacia significativa con una reducci贸n del 78% en la carga de tratamiento en comparaci贸n con el tratamiento proyectado con aflibercept.
Los resultados clave incluyen ganancias sostenidas en la agudeza visual de +9.7 letras y una mejora anat贸mica con una reducci贸n del grosor de la retina central (CST) de -174 碌m. El tratamiento fue bien tolerado sin inflamaci贸n intraocular observada. Notablemente, los pacientes que recibieron la dosis de Fase 3 requirieron solo 1.6 inyecciones suplementarias promedio en comparaci贸n con 3.7 para dosis m谩s bajas y 7.0 proyectadas para aflibercept.
Tanto la FDA como la EMA han acordado que un 煤nico ensayo cl铆nico de Fase 3 ser铆a aceptable para la presentaci贸n regulatoria de 4D-150 en DME, agilizando el camino hacia una posible aprobaci贸n.
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4D Molecular Therapeutics (NASDAQ:FDMT) a annonc茅 des r茅sultats positifs 脿 60 semaines de l'essai clinique SPECTRA 茅valuant le 4D-150 chez des patients atteints d'艙d猫me maculaire diab茅tique (DME). La dose de phase 3 (3E10 vg/艙il) a d茅montr茅 une efficacit茅 significative avec une r茅duction de 78 % de la charge de traitement par rapport au traitement projet茅 par aflibercept.
Les r茅sultats cl茅s incluent des gains soutenus d'acuit茅 visuelle de +9,7 lettres et une am茅lioration anatomique avec une r茅duction de l'茅paisseur fov茅olaire centrale (CST) de -174 碌m. Le traitement a 茅t茅 bien tol茅r茅 sans inflammation intraoculaire observ茅e. Notamment, les patients recevant la dose de phase 3 n'ont n茅cessit茅 en moyenne que 1,6 injections suppl茅mentaires contre 3,7 pour les doses inf茅rieures et 7,0 projet茅es pour aflibercept.
La FDA et l'EMA se sont align茅es sur le fait qu'un essai clinique de phase 3 unique serait acceptable pour la soumission r茅glementaire du 4D-150 dans le DME, simplifiant ainsi la voie vers une approbation potentielle.
4D Molecular Therapeutics (NASDAQ:FDMT) gab positive 60-Wochen-Ergebnisse der SPECTRA-Studie bekannt, die 4D-150 bei Patienten mit diabetischem Makula枚dem (DME) untersucht. Die Phase-3-Dosis (3E10 vg/Auge) zeigte eine signifikante Wirksamkeit mit einer 78%igen Reduktion der Behandlungsbelastung im Vergleich zur prognostizierten Aflibercept-Behandlung.
Wichtige Ergebnisse umfassen anhaltende Sehsch盲rfegewinne von +9,7 Buchstaben und anatomische Verbesserungen mit einer Verringerung der zentralen Netzhautdicke (CST) um -174 碌m. Die Behandlung wurde gut vertragen, ohne dass eine intraokulare Entz眉ndung beobachtet wurde. Bemerkenswert ist, dass Patienten mit der Phase-3-Dosis nur im Durchschnitt 1,6 zus盲tzliche Injektionen ben枚tigten, verglichen mit 3,7 bei niedrigeren Dosen und 7,0 prognostiziert bei Aflibercept.
Sowohl die FDA als auch die EMA haben sich darauf geeinigt, dass eine einzelne Phase-3-Studie f眉r die Zulassung von 4D-150 bei DME akzeptabel ist, was den Weg zur m枚glichen Zulassung erleichtert.
- Phase 3 dose achieved 78% reduction in treatment burden vs. standard aflibercept therapy
- Strong efficacy with +9.7 letters gain in visual acuity and -174 碌m CST reduction
- Excellent safety profile with no intraocular inflammation at any timepoint
- FDA and EMA agreement for streamlined approval pathway requiring only one Phase 3 trial
- Clear dose-dependent response with Phase 3 dose showing superior results
- Relatively small patient sample size (n=22) in the trial
- Two patients on lower dose missed >50% of study visits and were not evaluable
Insights
4D-150 shows strong efficacy for DME with 78% fewer injections needed vs. standard care, plus EMA regulatory approval for streamlined trials.
The 60-week SPECTRA trial results for 4D-150 in diabetic macular edema (DME) demonstrate remarkable efficacy and safety profiles that could revolutionize treatment. At the Phase 3 dose (3E10 vg/eye), patients experienced a substantial +9.7 letter improvement in visual acuity and -174 渭m reduction in central subfield thickness, indicating significant anatomical improvement.
What's particularly groundbreaking is the 78% reduction in treatment burden compared to standard aflibercept injections. The Phase 3 dose required only 1.6 supplemental injections versus the projected 7.0 for standard care, with 4 of 9 patients remaining injection-free after initial treatment. This represents a paradigm shift for DME patients, who typically require frequent injections over their lifetime.
The safety profile is exceptionally clean - zero cases of intraocular inflammation across all timepoints and dose levels, with no serious adverse events reported. The absence of inflammation is particularly noteworthy for a gene therapy, as inflammatory responses have historically been a concern.
The regulatory pathway has been significantly de-risked with both FDA and EMA alignment on a single Phase 3 trial for DME approval. This streamlined approach could accelerate the timeline to market while reducing development costs. For DME patients, particularly working-age individuals who struggle with treatment adherence, a single-administration therapy that provides durable results would address a critical unmet need in ophthalmology.
- 4D-150 continues to be well tolerated with no intraocular inflammation observed at any timepoint or dose level
- 4D-150 demonstrated durable and dose-dependent clinical activity with sustained gains in visual acuity and anatomic control
- Phase 3 dose (3E10 vg/eye) achieved clinically meaningful
78% reduction in treatment burden vs. projected on-label aflibercept 2mg Q8W - EMA aligned with proposed single Phase 3 clinical trial being acceptable for regulatory submission for 4D-150 in DME, consistent with previously announced alignment with FDA
EMERYVILLE, Calif., July 31, 2025 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics聽(Nasdaq: FDMT, 4DMT or the Company), a leading late-stage biotechnology company advancing durable and disease-targeted therapeutics with potential to transform treatment paradigms and provide unprecedented benefits to patients, today announced positive results from the SPECTRA clinical trial evaluating 4D-150 in patients with diabetic macular edema (DME), and alignment with the European Medicines Agency (EMA) on a registrational pathway for 4D-150 in DME. The data, which included both the 52-week primary endpoint and 60-week analyses, were presented by David Almeida, M.D., MBA, Ph.D., Erie Retina Research in an oral presentation titled 鈥淚nterim Results from the SPECTRA Phase 2a Clinical Trial Evaluating Intravitreal 4D-150 in Adults with Diabetic Macular Edema鈥� at the 43rd Annual American Society of Retina Specialists (ASRS) Scientific Meeting.
鈥淭he positive results from the SPECTRA trial demonstrate the tolerability and consistent, durable clinical activity of 4D-150 in DME, highlighting the potential for the product candidate to become a backbone therapy that can dramatically reduce treatment burden compared to the labeled regimen of standard-of-care aflibercept 2mg every eight weeks,鈥� said David Almeida, M.D., MBA, Ph.D. 鈥�4D-150 has the potential to fundamentally transform the treatment of DME by reducing treatment burden with a product that has adherence by design, while providing meaningful, lasting vision improvement. This is especially important in DME, which frequently occurs in a working-age population.鈥�
Clinical Trial Design & Interim Data from 4D-150 SPECTRA Clinical Trial (Data Cutoff of May 2, 2025):
- Objectives: Evaluate safety and tolerability, and identify dose level for further evaluation
- Study Population:
- Enrolled patients with high disease activity as measured by central subfield thickness (CST)
- 22 patients enrolled across 3 dose levels:
- 3E10 vg/eye (n=9) (Phase 3 dose)
- Lower doses (1E10 vg/eye, n=12; 5E9 vg/eye, n=1)
- 2 patients dosed with 1E10 vg/eye missed >
50% of study visits and were considered not evaluable for injection burden or other efficacy parameters
- 2 patients dosed with 1E10 vg/eye missed >
- Safety Results Through 60 Weeks (n=22):
- 4D-150 was well tolerated with no intraocular inflammation at any timepoint
- No subjects required modification to the topical corticosteroid regimen, and all patients are currently off corticosteroids
- No hypotony, endophthalmitis, vasculitis, choroidal effusions or retinal artery occlusions were reported
- Mean intraocular pressure was within normal limits
- 4D-150 was well tolerated with no intraocular inflammation at any timepoint
- Efficacy Results Through 60 Weeks:
- Utilized stringent supplemental aflibercept criteria to maximize patient safety while assessing initial clinical activity
- Phase 3 Dose:
- Sustained gain of best corrected visual acuity (BCVA) of +9.7 letters
- Sustained improvement in anatomic control, with reduction of CST, as measured by optical coherence tomography (OCT), of -174 碌m
- Supplemental Injections: Post-aflibercept loading doses (3), patients treated with Phase 3 dose required substantially fewer supplemental injections compared to patients receiving lower doses or projected on-label aflibercept 2mg Q8W (expected Phase 3 comparator):
- Mean supplemental injections per patient:
- Phase 3 dose: 1.6
- Lower doses: 3.7
- Projected on-label aflibercept 2mg Q8W: 7.0
- Dose response observed for Phase 3 dose vs. lower doses (
58% fewer injections) - Phase 3 dose demonstrated a reduction of
78% vs. projected on-label aflibercept 2mg Q8W
- 0-1 injections:
- 5 of 9 overall (Phase 3 dose) vs. 2 of 11 (lower doses)
- Injection-free:
- 4 of 9 overall (Phase 3 dose) vs. 1 of 11 overall (lower doses)
- Mean supplemental injections per patient:
4D-150 DME Phase 3 Regulatory Update
- Following U.S. Food and Drug Administration (FDA) alignment, , EMA is also aligned that a single Phase 3 clinical trial, based on data generated to date for 4D-150 in both the SPECTRA and PRISM clinical trials combined with data from the two planned Phase 3 clinical trials in the 4FRONT wet age-related macular degeneration (wet AMD) program, would be acceptable as the basis for a marketing authorization application (MAA) submission for 4D-150 in DME
鈥淭he consistency of dose response, safety and efficacy data we鈥檝e seen with 4D-150 across all patients evaluated in both DME and wet AMD reinforces our belief that 4D-150 has the potential to become a true backbone therapy, and may significantly improve the lives of millions of patients living with retinal vascular disease,鈥� said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT. 鈥淲ith alignment from both FDA and EMA for a single Phase 3 DME trial to complement the 4FRONT wet AMD program, we believe we have a clear streamlined path to bring 4D-150 to patients with high unmet need in two of the most prevalent blinding retinal vascular diseases.鈥�
About 4D-150
4D-150 is an investigational agent with the potential to become a backbone therapy that is designed to provide multi-year sustained delivery of anti-VEGF (aflibercept and anti-VEGF-C) from the retina with a single, safe, intravitreal injection. 4D-150 utilizes the Company鈥檚 customized and evolved intravitreal vector, R100, which was invented at 4DMT through the Company鈥檚 proprietary Therapeutic Vector Evolution platform. 4D-150 is being developed for wet AMD and DME, which both affect millions of patients globally, with the goal of freeing patients from burdensome injections while preserving vision.
About DME
DME, or diabetic macular edema, is a complication of diabetic retinopathy and is a highly prevalent disease with significant unmet medical need and poor treatment adherence. It is estimated that there are approximately one million individuals with DME in the U.S. according to published data. DME is characterized by inflammation swelling in the macula due to leakage from blood vessels, which can lead to vision loss. DME is typically treated with intravitreal anti-VEGF agents administered approximately every 4-12 weeks, although patient compliance with therapy is poor and results in high unmet medical need.
About 4DMT
4DMT is a leading late-stage biotechnology company advancing durable and disease-targeted therapeutics with potential to transform treatment paradigms and provide unprecedented benefits to patients. The Company鈥檚 lead product candidate 4D-150 is designed to be a backbone therapy forming the foundation of treatment of blinding retinal vascular diseases by providing multi-year sustained delivery of anti-VEGF (aflibercept and anti-VEGF-C) with a single, safe, intravitreal injection, which substantially reduces the treatment burden associated with current bolus injections. The Company鈥檚 lead indication for 4D-150 is wet age-related macular degeneration, which is currently in Phase 3 development, and second indication is diabetic macular edema. The Company鈥檚 second product candidate is 4D-710, which is the first known genetic medicine to demonstrate successful delivery and expression of the CFTR transgene in the lungs of people with cystic fibrosis after aerosol delivery. 4D Molecular Therapeutics鈩�, 4DMT鈩�, Therapeutic Vector Evolution鈩�, and the 4DMT logo are trademarks of 4DMT.
All of the Company鈥檚 product candidates are in clinical or preclinical development and have not yet been approved for marketing by the U.S. Food and Drug Administration or any other regulatory authority. No representation is made as to the safety or effectiveness of the Company鈥檚 product candidates for the therapeutic uses for which they are being studied.
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Forward-Looking Statements:
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, implied and express statements regarding the therapeutic potential and clinical development plans for 4D-150 and interactions with FDA and EMA. The words 鈥渕ay,鈥� 鈥渕ight,鈥� 鈥渨ill,鈥� 鈥渃ould,鈥� 鈥渨ould,鈥� 鈥渟hould,鈥� 鈥渆xpect,鈥� 鈥減lan,鈥� 鈥渁nticipate,鈥� 鈥渋ntend,鈥� 鈥渂elieve,鈥� 鈥渆xpect,鈥� 鈥渆stimate,鈥� 鈥渟eek,鈥� 鈥減redict,鈥� 鈥渇uture,鈥� 鈥減roject,鈥� 鈥減otential,鈥� 鈥渃ontinue,鈥� 鈥渢arget鈥� and similar words or expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including risks and uncertainties that are described in greater detail in the section entitled 鈥淩isk Factors鈥� in 4D Molecular Therapeutics鈥� most recent Quarterly Report on Form 10-Q filed on May 8, 2025 as well as any subsequent filings with the Securities and Exchange Commission. In addition, any forward-looking statements represent 4D Molecular Therapeutics' current views and should not be relied upon as representing its views as of any subsequent time. 4D Molecular Therapeutics explicitly disclaims any obligation to update any forward-looking statements. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements.
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FAQ
What were the key results from 4D-150's SPECTRA trial in DME patients?
How safe is FDMT's 4D-150 treatment based on the 60-week data?
What is the regulatory pathway for 4D-150 in DME?
How many supplemental injections did patients need with 4D-150's Phase 3 dose?
What percentage of Phase 3 dose patients remained injection-free?
